Lysosomal acid lipase (LAL) is a naturally occurring human enzyme involved in the metabolism of certain lipids, including cholesteryl esters and triglycerides. Certain diseases are caused by or exacerbated by a lack of LAL in the body, leading to serious and even life-threatening accumulations of lipids in major organs.
In addition to orphan indications, LAL could be potential used in treating arterial lipid plaque deposits, such as those that occur in atherosclerosis, without the necessity of surgery or other interventions, according to the developer.
While the orphan diseases are principally the result of genetic disorders in LAL synthesis or regulation, cardiovascular deposits occur in substantially larger numbers of patients and could be the result of genetics as well as environmental and lifestyle factors, such as diet.
Large Scale Bio Corp obtained a worldwide, exclusive license to all uses of LAL from Cincinnati Children’s Hospital Medical Center. The company, in conjunction with the Cincinnati Children’s group, is currently in preclinical development of its experimental LAL candidate.